Table 1 FDA-approved indications for included treatments
From: Patients’ access to rare neuromuscular disease therapies varies across US private insurers
International nonproprietary name | Brand name | FDA-approved indication |
|---|---|---|
Onasemnogene abeparvovec—xioi | Zolgensma | ZOLGENSMA (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene Limitation of Use The safety and effectiveness of repeat administration of ZOLGENSMA have not been evaluated The use of ZOLGENSMA in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated |
Nusinersen | Spinraza | SPINRAZA is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients |
Edaravone | Radicava | RADICAVA is indicated for the treatment of amyotrophic lateral sclerosis (ALS) |
Eteplirsen | Exondys 51 | EXONDYS 51 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51 [see Clinical Studies (14)] A clinical benefit of EXONDYS 51 has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials |